The work of researchers from the CHU Sainte-Justine has made it possible to identify a promising molecule in the treatment of Duchenne muscular dystrophy, without the undesirable side effects of current therapy.
Duchenne muscular dystrophy (DMD) is a rare genetic disorder and is characterized by progressive muscle degeneration.
The administration of glucocorticoids in childhood is the standard treatment, but these steroids can also have the effect of directly attacking the muscles and reducing their healing capacity.
“On the one hand, we protect our muscle by reducing inflammation with glucocorticoids, but on the other hand we harm it because we prevent it from regenerating normally”, summarized Professor Nicolas Dumont, who is researcher at CHU Sainte-Justine and associate professor at the School of Rehabilitation of the University of Montreal.
The relative toxicity of glucocorticoids can also cause significant side effects such as osteoporosis, growth retardation in children, or acute anxiety.
DMD is characterized by fragile muscles that tear at the slightest contraction, setting off a vicious cycle of injury and healing. Chronic inflammation that attacks healthy tissue and helps destroy more muscle sets in over time.
Glucocorticoids can fight this inflammation, but at the cost of significant unwanted side effects. Professor Dumont and his colleagues have identified a molecule, Resolvin-D2, which is a very effective anti-inflammatory. And unlike glucocorticoids, it also stimulates the activity of muscle stem cells which are responsible for healing the muscles.
“When compared to each other in preclinical animal models, (Resolvin-D2) is more effective than glucocorticoids,” said Dumont. It is a molecule that is endogenous, so it is a molecule that is made by the body. “
The goal of the project, he continues, is not necessarily to cure the disease, but rather to improve the treatment of patients to offer them a better quality of life.
Researchers are continuing their work to create a more stable Resolvin-D2 molecule, which degrades less quickly, and which could be administered orally.
DMD affects one in 4000 boys. The first symptoms of the disease appear around the age of 3 to 5 years, and as the child grows, there is an irreversible loss of muscle function. Patients with it rarely survive beyond the age of 30.
The results of this study are published in the journal Nature Communications.